Gene therapy shows promise for rare eye disease that causes blindness
NCT ID NCT03293524
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This Phase 3 trial tested a gene therapy called GS010 in 98 people with Leber hereditary optic neuropathy (LHON), a genetic condition that leads to rapid vision loss. Participants received injections of the therapy or a placebo into both eyes. The study measured changes in vision over 1.5 years, with follow-up up to 5 years. The goal was to see if GS010 could improve or stabilize eyesight in those with vision loss for less than a year.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
GS010 (lenadogene nolparvovec) gene therapy
What this could lead to
If successful, this gene therapy could slow or partially reverse vision loss in people with a specific form of Leber hereditary optic neuropathy.
What could go wrong
This is a Phase 3 trial, but results may not apply to all patients. Gene therapy carries risks like injection-related complications, and long-term effects are still being studied.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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CHNO Les Quinze Vingts
Paris, Paris, 75012, France
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Department of Ophthalmology, Icahn School of Medicine at Mount Sinai
New York, New York, 10029, United States
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Departments of Neurology and Ophthalmology, Wills Eye Hospital and Thomas Jefferson University
Philadelphia, Pennsylvania, 19107, United States
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Doheny Eye Center UCLA Pasadena
Pasadena, California, 91105, United States
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Emory Healthcare - The Emory Clinic
Atlanta, Georgia, 30322, United States
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Hospital Universitario Ramon y Cajal
Madrid, 28034, Spain
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IRCCS Istituto delle Scienze Neurologiche di Bologna (ISNB) Clinica Neurologica
Bologna, 40139, Italy
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Massachusetts Eye and Ear Infirmary
Boston, Massachusetts, 02114, United States
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Moorfields Eye Hospital
London, Greater London, EC1V 2PD, United Kingdom
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Taipei Veterans General Hospital
Taipei, 11217, Taiwan
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Universitair Ziekenhuis Gent
Ghent, 9000, Belgium
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University of Colorado Health Eye Center
Aurora, Colorado, 80045, United States
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Vanderbilt Eye Institute
Nashville, Tennessee, 37232, United States